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Pridopidine Phase 3 Study in Huntington's Disease (PRECISE-HD)

2026年9月14日 更新者：Prilenia

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Huntington's Disease (HD)

The goal of this clinical trial is to learn if pridopidine can slow the clinical decline of Huntington's Disease (HD) in adult participants. It will also inform about the safety of pridopidine.

The main questions the study aims to answer are:

Does pridopidine slow the overall worsening of HD over 1 year? Does pridopidine slow the worsening of specific aspects of HD over 1 year, namely the clinical progression, the ability to perform daily life activities (functional capacity), the mind's ability to process information (cognition), working of the muscles (motor function), and quality of life?

Researchers will compare the drug pridopidine to a placebo (a look-alike substance that contains no drug) to see if pridopidine works better than placebo to treat HD. During the first year of the study, participants will have the same chance to receive either pridopidine or placebo.

Participants will:

Take 1 pridopidine or placebo capsule twice daily for 12 months. Visit the clinic 6 times within 1 year for checkups and tests.

All participants who complete this 1-year placebo-controlled study period will roll over into an additional 2-year study period during which all participants will receive pridopidine treatment, including participants who had received placebo during the first year. During this additional 2-year treatment period participants will visit the clinic a total of 6 times for checkups and tests.

研究概览

地位

招聘中

条件

干预/治疗

详细说明

This Phase 3 study consists of two study periods, a randomized, double-blind, placebo-controlled 1-year study period and a 2-year open-label extension (OLE) period. The study assesses the effect of pridopidine 45 mg twice daily on HD in participants who are not using antidopaminergic medications (ADMs) at study start. Main objectives of the study will be to assess the effect of pridopidine on clinical progression of HD within 1 year and to evaluate the effect of pridopidine on functional capacity, cognition, motor function, quality of life (QoL), and speech in participants with HD within 1 year.

The target population in this study are adult participants (age 23-65, inclusive) with adult-onset HD (onset of signs and symptoms and a diagnosis ≥21 years of age). Eligible participants have not been using ADMs for at least 6 months prior to the screening visit. In case ADM treatment is deemed necessary by the treating physician during the study, specific ADMs and doses may be initiated. Female participants who are pregnant, planning to become pregnant or breastfeeding are not allowed to enter the study.

The treatment period will start with a 2-week titration period of once-daily treatment (1 capsule of pridopidine or placebo taken orally in the morning). From Day 15 onwards, all participants will take the study drug twice daily: in the morning and in the afternoon. Treatment will continue for an additional 50 weeks.

The placebo-controlled study period includes 6 in-clinic visits and 5 telephone visits (safety calls) during the 1-year treatment period.

The placebo-controlled study period will be followed by a 2-year OLE period. During the OLE period, all eligible participants will receive pridopidine. The OLE will consist of a 2-year treatment period, including an initial 2-week (re)titration period for all participants at pridopidine once daily and a maintenance period of 102 weeks with pridopidine twice daily. The blinding of the original treatment assignment will be maintained for all participants and study personnel during the OLE period.

A total of 6 in-clinic visits and 6 telephone visits (safety calls) are planned during the 2-year OLE period.

研究类型

介入性

注册 (估计的)

400

阶段

  • 第三阶段

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

学习地点

  • 加拿大
    • Alberta
      • Calgary、Alberta、加拿大
        • 尚未招聘
        • University Of Calgary
      • Edmonton、Alberta、加拿大
        • 尚未招聘
        • University of Alberta
    • British Columbia
      • Vancouver、British Columbia、加拿大
        • 尚未招聘
        • University of British Columbia
    • Ontario
      • Toronto、Ontario、加拿大
        • 尚未招聘
        • North York General Hospital
    • Quebec
      • Montreal、Quebec、加拿大
        • 尚未招聘
        • Centre hospitalier de l'Université de Montréal (CHUM)
  • 德国
      • Aachen、德国
        • 尚未招聘
        • Universitaetsklinikum Aachen AöR
      • Bochum、德国
        • 尚未招聘
        • Katholisches Klinikum Bochum gGmbH
      • Erlangen、德国
        • 尚未招聘
        • Universitätsklinikum Erlangen
      • Münster、德国
        • 尚未招聘
        • George-Huntington-Institut GmbH
      • Taufkirchen、德国
        • 尚未招聘
        • Isar Amper Klinikum
      • Ulm、德国
        • 尚未招聘
        • Universitaetsklinikum Ulm AöR
  • 意大利
      • Bologna、意大利
        • 尚未招聘
        • Azienda Unita Sanitaria Locale Di Bologna
      • Milan、意大利
        • 尚未招聘
        • IRCCS Istituto Auxologico Italiano
      • Naples、意大利
        • 尚未招聘
        • Azienda Ospedaliera Universitaria Federico II di Napoli
      • Padova、意大利
        • 尚未招聘
        • Azienda Ospedale Università di Padova
      • Roma、意大利
        • 尚未招聘
        • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
      • San Giovanni Rotondo、意大利
        • 尚未招聘
        • Casa Sollievo Della Sofferenza
  • 捷克语
      • Prague、捷克语
        • 尚未招聘
        • Vseobecna fakultni nemocnice v Praze
  • 比利时
      • Anderlecht、比利时
        • 尚未招聘
        • Hopital Erasme
      • Woluwe-Saint-Lambert、比利时
        • 尚未招聘
        • Cliniques universitaires Saint-Luc
  • 法国
      • Angers、法国
        • 尚未招聘
        • Centre Hospitalier Universitaire d'Angers
      • Créteil、法国
        • 尚未招聘
        • CHU Henri Mondor (APHP)
      • La Tronche、法国
        • 尚未招聘
        • Centre Hospitalier Universitaire Grenoble Alpes
      • Marseille、法国
        • 尚未招聘
        • Centre Hospitalier Regional De Marseille
  • 波兰
      • Krakow、波兰
        • 尚未招聘
        • Krakowska Akademia Neurologii Sp. z o.o.
      • Warsaw、波兰
        • 尚未招聘
        • Wojskowy Instytut Medycyny Lotniczej
  • 美国
    • Alabama
      • Birmingham、Alabama、美国、35294
        • 尚未招聘
        • The University of Alabama at Birmingham
    • California
      • La Jolla、California、美国、92037
        • 招聘中
        • University of California, San Diego
        • 接触：
      • Los Angeles、California、美国、90095
        • 尚未招聘
        • UCLA Medical Center
      • Sacramento、California、美国、95817
        • 尚未招聘
        • UC Davis Medical Center
    • District of Columbia
      • Washington D.C.、District of Columbia、美国、20007
        • 尚未招聘
        • Georgetown University
    • Florida
      • Boca Raton、Florida、美国、33486
        • 尚未招聘
        • University of Miami-U Health Boca Raton
      • Gainesville、Florida、美国、32608
        • 尚未招聘
        • University of Florida (Norman Fixel Institute for Neurological Diseases)
    • Illinois
      • Chicago、Illinois、美国、60611
        • 尚未招聘
        • Northwestern University
      • Chicago、Illinois、美国、60612
        • 尚未招聘
        • Rush University Medical Center
    • Kansas
      • Kansas City、Kansas、美国、66103
        • 尚未招聘
        • University of Kansas Medical Center
      • Wichita、Kansas、美国、67226
        • 招聘中
        • Hereditary Neurological Disease Centre
        • 接触：
    • Maryland
      • Baltimore、Maryland、美国、60612
        • 尚未招聘
        • Johns Hopkins University
    • Massachusetts
      • Charlestown、Massachusetts、美国、02129
        • 尚未招聘
        • Massachusetts General Hospital
    • Minnesota
      • Golden Valley、Minnesota、美国、55427
        • 尚未招聘
        • Struthers Parkinson's Center / Health Partners Institute
    • New York
      • New York、New York、美国、10032
        • 尚未招聘
        • Columbia University
    • North Carolina
      • Durham、North Carolina、美国、27705
        • 尚未招聘
        • Duke University
    • Ohio
      • Cincinnati、Ohio、美国、45219
        • 尚未招聘
        • University of Cincinnati
      • Cleveland、Ohio、美国、44195
        • 尚未招聘
        • Cleveland Clinic
      • Columbus、Ohio、美国、43221
        • 招聘中
        • The Ohio State University
    • Oregon
      • Portland、Oregon、美国、97239
        • 尚未招聘
        • Oregon Health and Science University (OHSU)
    • Tennessee
      • Nashville、Tennessee、美国、37212
        • 招聘中
        • Vanderbilt University Medical Center
    • Texas
      • Houston、Texas、美国、77030
        • 尚未招聘
        • UT Health, Houston, McGovern Medical School
    • Vermont
      • Burlington、Vermont、美国、05401
        • 尚未招聘
        • University of Vermont Medical Center
    • Washington
      • Kirkland、Washington、美国、98034
        • 尚未招聘
        • EvergreenHealth Research Department
      • Seattle、Washington、美国、98195
        • 招聘中
        • University of Washington
  • 英国
      • Aberdeen、英国
        • 尚未招聘
        • NHS Grampian
      • Birmingham、英国
        • 尚未招聘
        • Birmingham and Solihull Mental Health Foundation NHS Trust
      • Cardiff、英国
        • 尚未招聘
        • Cardiff University
      • Liverpool、英国
        • 尚未招聘
        • The Walton Centre NHS Foundation Trust
      • Newcastle upon Tyne、英国
        • 尚未招聘
        • CNTW NHS Foundation Trust
  • 荷兰
      • Leiden、荷兰
        • 尚未招聘
        • Leids Universitair Medisch Centrum (LUMC)
  • 葡萄牙
      • Coimbra、葡萄牙
        • 尚未招聘
        • Unidade Local De Saude De Coimbra E.P.E
      • Lisbon、葡萄牙
        • 尚未招聘
        • Unidade Local De Saude De Santa Maria E.P.E.
      • Porto、葡萄牙
        • 尚未招聘
        • Unidade Local De Saude De Santo Antonio E.P.E.
      • Torres Vedras、葡萄牙
        • 尚未招聘
        • CNS Saude Lda.
  • 西班牙
      • Barakaldo、西班牙
        • 尚未招聘
        • Hospital Universitario de Cruces
      • Barcelona、西班牙
        • 尚未招聘
        • Hospital de La Santa Creu i Sant Pau
      • Madrid、西班牙
        • 尚未招聘
        • Hospital Universitario Ramon y Cajal
      • Oviedo、西班牙
        • 尚未招聘
        • Hospital Universitario Central de Asturias
      • Seville、西班牙
        • 尚未招聘
        • Hospital Universitario Virgen del Rocío

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人
  • 年长者

接受健康志愿者

不

描述

Inclusion Criteria:

  • Adult-onset HD (onset of signs and symptoms and a clinical diagnosis at ≥21 years of age).
  • A diagnosis based on clinical features and the presence of ≥40 CAG repeats in the huntingtin (HTT) gene confirmed by historical laboratory quanitified results or by a diagnostic test at Screening.
  • Diagnostic confidence level (DCL) of 4 (DCL=4 unequivocal motor signs, ≥99% confidence) on the standardized motor exam Total Motor Score (TMS).
  • Total Functional Capacity (TFC) score of ≥7 at Screening and Baseline.
  • Cytosine-Adenine-Guanine (CAG)-Age Product (CAP)100 score ≥95 at Screening.
  • Independence Scale (IS) score ≤90% at Screening.
  • Total Motor Score (TMS) of ≥20 at Screening and Baseline.
  • Not using ADMs (VMAT2i and neuroleptics/antipsychotics) for at least 6 months prior to Screening visit. Importantly, it is not encouraged to discontinue the participants' ADM treatment solely for enrollment in the current trial.

Exclusion Criteria:

  • Clinically significant cardiovascular disease (e.g. QTcF >450 msec [males] or >470 msec [females], arrhythmias, uncontrolled atrial fibrillation, or congenital long QT syndrome), seizure history ≤5 years, significant neurological disorders (e.g. intracranial pathology or cerebrovascular events), active/recent malignancy (unless localized and resolved), or any serious or uncontrolled systemic disease (e.g. hepatic, renal, respiratory, endocrine, infectious [HBV, HCV, HIV], or psychiatric) that may pose safety risk or interfere with participation.
  • Severe hepatic or renal impairment.
  • Any mutant huntingtin (mHTT) lowering therapy in the past year.
  • Medications that prolong QT interval, taken within 4 weeks of the baseline visit.
  • Use of pridopidine within 6 weeks or 5 half-lives before the screening visit.
  • Previous participation in intracranial gene therapy study.
  • Laboratory values that fall outside of the central laboratory's reference range at Screening and are considered clinically significantly abnormal by the Investigator and affect the participant's suitability to participate in the study or put the participant at risk if he/she enters the study in the Investigator's opinion.
  • Female participants who are pregnant, planning to become pregnant or breastfeeding.

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：并行分配
  • 屏蔽：四人间

武器和干预

参与者组/臂
干预/治疗
实验性的：Pridopidine
During the titration period (2 weeks), pridopidine 45 mg capsules will be taken orally once daily (in the morning) for 2 weeks. Afterwards, pridopidine 45 mg capsules will be taken orally twice daily (in the morning and in the afternoon; 7-10 hours apart) for 50 weeks.
药品：普利多匹定
普利多匹定硬明胶胶囊
安慰剂比较：Placebo
During the titration period (2 weeks), matched placebo hard gelatin capsules will be taken once daily (in the morning) for 2 weeks. Afterwards, placebo capsules will be taken twice daily (in the morning and in the afternoon; 7-10 hours apart) for 50 weeks.
药品：Placebo
Matched placebo hard gelatin capsule

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Change in Composite Unified Huntington's Disease Rating Scale (cUHDRS)
大体时间：Change from Baseline to Week 52

cUHDRS is a combined score to assess disease progression in HD. It includes TFC, TMS, SDMT, and SWR:

Total Functional Capacity (TFC) tests the capacity to maintain daily living, finances, care level, occupation. Higher score = better outcome. Best score=13. Worst score=0.

Total Motor Score (TMS) assesses motor features (oculomotor, dysarthria, chorea, dystonia, gait, postural stability). Higher score = worse outcome. Best score=0. Worst score= 124.

Stroop Word Reading (SWR) measures attention and mental flexibility. Participant reads names of colors printed in black ink. Scores reflect correct responses in 45 sec. Higher score = better outcome. Best score=100. Worst score=0.

Symbol Digit Modalities Test (SDMT) tests psychomotor speed and working memory. Participant has 90 sec to match numbers with symbols. Scores = correct answers in 90 sec. Higher score = better outcome. Best score=120. Worst score=0.

Total integrated cUHDRS scale range: -7.6 to 24.8. The higher, the better.
Change from Baseline to Week 52

次要结果测量

结果测量
措施说明
大体时间
Change in Total Functional Capacity (TFC) score
大体时间：Change from Baseline to Week 52
Total Functional Capacity (TFC) tests the capacity to maintain domestic chores, activities of daily living, finances, care level, and occupation. Scores from 0 - 13. Higher score = better outcome.
Change from Baseline to Week 52
Change in Quantitative Motor (Q-Motor) Finger Tapping Inter-Onset Interval (FT-IOI) mean
大体时间：Change from Baseline to Week 52
Quantitative (Q)-motor is a clinical assessment of fine motor skills that are crucial for daily activities. It is based on the application of pre-calibrated and temperature-controlled force transducers and 3-dimensional position sensors. Higher score = worse outcome.
Change from Baseline to Week 52
Change in Stroop Word Reading (SWR) score
大体时间：Change from Baseline to Week 52
Stroop Word Reading (SWR) measures attention and mental flexibility. Participant reads names of colors printed in black ink. Scores reflect correct responses in 45 sec. Higher score = better outcome.
Change from Baseline to Week 52

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Prilenia

合作者

Ferrer Internacional S.A.

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2026年7月1日

初级完成 (估计的)

2028年6月1日

研究完成 (估计的)

2030年6月1日

研究注册日期

首次提交

2026年5月20日

首先提交符合 QC 标准的

2026年5月20日

首次发布 (实际的)

2026年5月27日

研究记录更新

最后更新发布 (实际的)

2026年9月17日

上次提交的符合 QC 标准的更新

2026年9月14日

最后验证

2026年5月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • PL101-HD302/ FNP253-CT-2502
  • 2026-526093-16-00 (克蒂斯)

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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